| Therapeutic indication | Clinical outcome | Surrogate outcome |
|---|---|---|
| Allergy | Bulbar conjunctival injection and ocular itching (seasonal allergic conjunctivitis) | No drugs approved |
| Cancer | Duration of survival (metastatic colorectal cancer) | Proportion of patients who achieved complete response or partial response (Hodgkin lymphoma) |
| Cardiovascular | Time to first event of cardiovascular death, myocardial infarction and stroke (acute coronary syndromes) | Change from baseline in trough sitting diastolic blood pressure (hypertension) |
| Dermatology | Change in total inflammatory lesions in adults (rosacea) | No drugs approved |
| Genitourinary | Number of incontinence episodes per week (overactive bladder) | No drugs approved |
| Gastrointestinal | No emetic episode and no rescue medication within 24 hours (emetogenic chemotherapy) | No drugs approved |
| Gynecology | Percentage of women with a reduction in uterine bleeding (uterine fibroids) | At least one follicle ≥ 17 mm, pre-ovulatory estradiol serum level ≥ 109 pg/mL (400 pmol/L), and mid-luteal phase progesterone level ≥ 7.9 ng/mL (25 nmol/L) (follicular development) |
| Hematology | Annualized bleeding rate per patient (congenital factor IX deficiency) | Proportion of patients who experienced a hemoglobin response (anemia associated with chronic kidney disease) |
| Inborn error of metabolism | Proportion of patients alive and free of invasive ventilator support (Pompe disease) | Reduction in mean spleen volume (type 1 Gaucher disease) |
| Infectious disease, hepatitis | No drugs approved | Cell histology (hepatitis B) |
| Infectious disease, HIV | No drugs approved | Proportion of patients with a treatment response ≥ 1 log10 reduction in viral load (HIV) |
| Infectious disease, other | Improvement in respiratory symptoms (cystic fibrosis) | No drugs approved |
| Infectious disease, vaccine | Protecting against severe rotavirus gastroenteritis episodes (rotavirus gastroenteritis) | Ability to induce antibodies against viral hemagglutinin (influenza) |
| Metabolic disease, diabetes | No drugs approved | Change in glycosylated hemoglobin (type 2 diabetes) |
| Metabolic disease, other | No drugs approved | Percent change in serum low-density lipoprotein cholesterol concentration (Frederickson type IIa familial hyperlipidemia) |
| Miscellaneous | Time to onset of relief of symptoms of abdominal or facial attack (hereditary angioedema) | Complete or partial cytogenetic response (graft-versus-host disease) |
| Musculoskeletal | Pain intensity, patient’s global assessment of disease activity, and the total score of the Western Ontario and McMaster Universities questionnaire (osteoarthritis of knee) | No drugs approved |
| Neurology | Median percent reduction in seizure frequency (epilepsy) | Biochemical marker 8OH2’dG (Friedreich ataxia) |
| Ophthalmology | Proportion of patients who maintained vision (macular degeneration) | Change from baseline in anterior chamber cell grade (anterior uveitis) |
| Psychiatry | Positive and Negative Syndrome Scale total score (schizophrenia) | No drugs approved |
| Pulmonary hypertension | Time to first occurrence of morbidity or mortality event (pulmonary arterial hypertension) | Distance covered in 6-minute walking test (pulmonary hypertension) |
| Respirology | Annual rate of moderate and severe exacerbations (chronic obstructive lung disease) | Forced expiratory volume in 1 second and peak expiratory flow (asthma) |
| Rheumatology | Proportion of patients with an ACR20 response (rheumatoid arthritis) | No drugs approved |
Note: 8OH2’dG = 8-hydroxy-2'-deoxyguanosine, ACR20 = American College of Rheumatology 20% improvement response.